MedPage Today April 26, 2024
— One dose of fidanacogene elaparvovec proved noninferior to standard prophylactic factor IX
The FDA has approved a second gene therapy for adults with hemophilia B, drugmaker Pfizer announced on Friday.
The approval of fidanacogene elaparvovec (Beqvez) stipulates use in adult men with moderate to severe hemophilia B, according to Pfizer’s statement. Specifically, the approval limits use to patients currently treated with prophylactic factor IX, who have a history of life-threatening hemorrhage, or who have repeated serious spontaneous bleeding episodes, and who do not have neutralizing antibodies to the adeno-associated virus serotype Rh74var capsid.
The gene therapy enables patients with hemophilia B to produce factor IX, rather than treat the condition with intravenous infusions of the blood factor.
“Many people...
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